Efficient adenoviral vector transduction of human hematopoietic SCID- repopulating and long-term culture-initiating cells

Human Gene Therapy

Volumn 11, Issue 9, 2000, Pages 1313-1327

  Fan, Xiaolong ^a,b   Brun, Ann ^a   Segrén, Sverker ^a   Jacobsen, Sten E W ^a   Karlsson, Stefan ^a  

^a Department of Laboratory Medicine ^*  (Sweden)

^b LUND UNIVERSITY   (Sweden)

Author keywords

[No Author keywords available]

Indexed keywords

FLT3 LIGAND; GREEN FLUORESCENT PROTEIN; STEM CELL FACTOR; THROMBOPOIETIN; VIRUS VECTOR;

ARTICLE; CELL CYCLE; CELL POPULATION; CELL PROLIFERATION; CELL SURVIVAL; COLONY FORMING UNIT C; COMBINED IMMUNODEFICIENCY; CONTROLLED STUDY; HEMATOPOIETIC CELL; HUMAN; HUMAN CELL; NONHUMAN; NORMAL HUMAN; PROMOTER REGION; SIGNAL TRANSDUCTION;

ADENOVIRIDAE; ADP-RIBOSYL CYCLASE; ANIMALS; ANTIGENS, CD; ANTIGENS, CD34; ANTIGENS, CD38; ANTIGENS, DIFFERENTIATION; BONE MARROW CELLS; CELL DIVISION; CELL LINEAGE; CELL SURVIVAL; FETAL BLOOD; GENE EXPRESSION; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HUMANS; LUMINESCENT PROTEINS; MEMBRANE GLYCOPROTEINS; MEMBRANE PROTEINS; MICE; MICE, SCID; NAD+ NUCLEOSIDASE; STEM CELL FACTOR; THROMBOPOIETIN; TRANSDUCTION, GENETIC;

EID: 0034631341     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430340050032410     Document Type: Article

References (40)

1. BAJOCCHI, G., FELDMAN, S.H., CRYSTAL, R.G., and MASTRANGELI, A. (1993). Direct in vivo gene transfer to ependymal cells in the central nervous system using recombinant adenovirus vectors. Nature Genet. 3, 229-234. [See comments]
2. BHATIA, M., WANG, J.C.Y., KAPP, U., BONNET, D., and DICK, J.E. (1997). Purification of primitive human hematopoietic cells capable of repopulating immune-deficient mice. Proc. Natl. Acad. Sci. U.S.A. 94, 5320-5325.
3. BHATIA, M., BONNET, D., WU, D., MURDOCH, B., WRANA, J., GALLACHER, L., and DICK, J.E. (1999). Bone morphogenetic proteins regulate the developmental program of human hematopoietic stem cells. J. Exp. Med. 189, 1139-1148.
4. BORGE, O.J., RAMSFJELL, V., VEIBY, O.P., MURPHY, M.J., JR., LOK, S., and JACOBSEN, S.E. (1996). Thrombopoietin, but not erythropoietin promotes viability and inhibits apoptosis of multipotent murine hematopoietic progenitor cells in vitro. Blood 88, 2859-2870.
5. BORGE, O.J., RAMSFJELL, V., CUI, L., and JACOBSEN, S.E. (1997). Ability of early acting cytokines to directly promote survival and suppress apoptosis of human primitive CD34+CD38-bone marrow cells with multilineage potential at the single-cell level: Key role of thrombopoietin. Blood 90, 2282-2292.
6. BREGNI, M., SHAMMAH, S., MALAFFO, F., DI NICOLA, M., MILANESI, M., MAGNI, M., MATTEUCCI, P., RAVAGNANI, F., JORDAN, C.T., SIENA, S., and GIANNI, A.M. (1998). Adenovirus vectors for gene transduction into mobilized blood CD34+ cells. Gene Ther. 5, 465-472.
7. BYK, T., HADDADA, H., VAINCHENKER, W., and LOUACHE, F. (1998). Lipofectamine and related cationic lipids strongly improve adenoviral infection efficiency of primitive human hematopoietic cells. Hum. Gene Ther. 9, 2493-2502.
8. CASHMAN, J.D., LAPIDOT, T., WANG, J.C., DOEDENS, M., SHULTZ, L.D., LANSDORP, P., DICK, J.E., and EAVES, C.J. (1997). Kinetic evidence of the regeneration of multilineage hematopoiesis from primitive cells in normal human bone marrow transplanted into immunodeficient mice. Blood 89, 4307-4316.
9. CHEN, L., PULSIPHER, M., CHEN, D., SIEFF, C., ELIAS, A., FINE, H.A., and KUFE, D.W. (1996). Selective transgene expression for detection and elimination of contaminating carcinoma cells in hematopoietic stem cell sources. J. Clin. Invest. 98, 2539-2548. [See comments]
10. DAO, M.A., HANNUM, C.H., KOHN, D.B., and NOLTA, J.A. (1997). FLT3 ligand preserves the ability of human CD34+ progenitors to sustain long-term hematopoiesis in immune-deficient mice after ex vivo retroviral-mediated transduction. Blood 89, 446-456.
11. - cells. In preparation.
12. FASBENDER, A., ZABNER, J., CHILLON, M., MONINGER, T.O., PUGA, A.P., DAVIDSON, B.L., and WELSH, M.J. (1997). Complexes of adenovirus with polycationic polymers and cationic lipids increase the efficiency of gene transfer in vitro and in vivo. J. Biol. Chem. 272, 6479-6489.
13. FREY, B.M., HACKETT, N.R., BERGELSON, J.M., FINBERG, R., CRYSTAL, R.G., MOORE, M.A., and RAFII, S. (1998). High-efficiency gene transfer into ex vivo expanded human hematopoietic progenitors and precursor cells by adenovirus vectors. Blood 91, 2781-2792.
14. GERDES, J., LEMKE, H., BAISCH, H., WACKER, H.H., SCHWAB, U., and STEIN, H. (1984). Cell cycle analysis of a cell proliferation-associated human nuclear antigen defined by the monoclonal antibody Ki-67. J. Immunol. 133, 1710-1715.
15. GOTHOT, A., PYATT, R., McMAHEL, J., RICE, S., and SROUR, E.F. (1997). Functional heterogeneity of human CD34(+) cells isolated in subcompartments of the G0/G1 phase of the cell cycle. Blood 90, 4384-4393.
16. HADDADA, H., CORDIER, L., and PERRICAUDET, M. (1995). Gene therapy using adenovirus vectors. In The Molecular Repertoire of Adenoviruses, Vol. III: Biology and Pathogenesis. W. Doerfler and P. Böhm, eds. (Springer-Verlag, Berlin) pp. 297-304.
17. HAO, Q.L., SHAH, A.J., THIEMANN, F.T., SMOGORZEWSKA, E.M., and CROOKS, G.M. (1995). A functional comparison of CD34+ CD38-cells in cord blood and bone marrow. Blood 86, 3745-3753.
18. HAO, Q.L., THIEMANN, F.T., PETERSEN, D., SMOGORZEWSKA, E.M., and CROOKS, G.M. (1996). Extended long-term culture reveals a highly quiescent and primitive human hematopoietic progenitor population. Blood 88, 3306-3313.
19. HITT, M., BETT, A.J., ADDISON, C.L., PREVEC, L., and GRAHAM, F.L. (1995). Techniques for human adenovirus vector construction and characterization. In Methods in Molecular Genetics. K.W. Adolph, ed. (Academic Press, San Diego, CA) pp. 13-30.
20. HOWARD, D.S., RIZZIERRI, D.A., GRIMES, B., UPCHURCH, D., PHILLIPS, G.L., STEWART, A.K., YANNELLI, J.R., and JORDAN, C.T. (1999). Genetic manipulation of primitive leukemic and normal hematopoietic cells using a novel method of adenovirus-mediated gene transfer. Leukemia 13, 1608-1616.
21. JORDAN, C.T., YAMASAKI, G., and MINAMOTO, D. (1996). High-resolution cell cycle analysis of defined phenotypic subsets within primitive human hematopoietic cell populations. Exp. Hematol. 24, 1347-1355.
22. KARLSSON, S. (1991). Treatment of genetic defects in hematopoietic cell function by gene transfer. Blood 78, 2481-2492.
23. KIMURA, S., ROBERTS, A.W., METCALF, D., and ALEXANDER, W.S. (1998). Hematopoietic stem cell deficiencies in mice lacking c-Mpl, the receptor for thrombopoietin. Proc. Natl. Acad. Sci. U.S.A. 95, 1195-1200.
24. LAROCHELLE, A., VORMOOR, J., HANENBERG, H., WANG, J.C., BHATIA, M., LAPIDOT, T., MORITZ, T., MURDOCH, B., XIAO, X.L., KATO, I., WILLIAMS, D.A., and DICK, J.E. (1996). Identification of primitive human hematopoietic cells capable of repopulating NOD/SCID moues bone marrow: Implications for gene therapy. Nature Med. 2, 1329-1337.
25. LUENS, K.M., TRAVIS, M.A., CHEN, B.P., HILL, B.L., SCOLLAY, R., and MURRAY, L.J. (1998). Thrombopoietin, kit ligand, and flk2/flt3 ligand together induce increased numbers of primitive hematopoietic progenitors from human CD34+ Thy-1+Lin-cells with preserved ability to engraft SCID-hu bone. Blood 91, 1206-1215.
26. MORRISON, S.J., SHAH, N.M., and ANDERSON, D.J. (1997). Regulatory mechanisms in stem cell biology. Cell 88, 287-298.
27. NEERING, S.J., HARDY, S.F., MINAMOTO, D., SPRATT, S.K., and JORDAN, C.T. (1996). Transduction of primitive human hematopoietic cells with recombinant adenovirus vectors. Blood 88, 1147-1155.
28. PETZER, A.L., HOGGE, D.E., LANDSDORP, P.M., REID, D.S., and EAVES, C.J. (1996). Self-renewal of primitive human hematopoietic cells (long-term-culture-initiating cells) in vitro and their expansion in defined medium. Proc. Natl. Acad. Sci. U.S.A. 93, 1470-1474.
29. PIACIBELLO, W., SANAVIO, F., GARETTO, L., SEVERINO, A., BERGANDI, D., FERRARIO, J., FAGIOLI F., BERGER, M., and AGLIETTA, M. (1997). Extensive amplification and self-renewal of human primitive hematopoietic stem cells from cord blood. Blood 89, 2644-2653.
30. QIU, C., DE YOUNG, M.B., FINN, A., and DICHEK, D.A. (1998). Cationic liposomes enhance adenovirus entry via a pathway independent of the fiber receptor and alpha(v)-integrins. Hum. Gene. Ther. 9, 507-520.
31. RAMSFJELL, V., BORGE, O.J., CUI, L., and JACOBSEN, S.E. (1997). Thrombopoietin directly and potently stimulates multilineage growth and progenitor cell expansion from primitive (CD34+ CD38-) human bone marrow progenitor cells: Distinct and key interactions with the ligands for c-kit and flt3, and inhibitory effects of TGF-beta and TNF-alpha. J. Immunol. 158, 5169-5177.
32. ROE, T., REYNOLDS, T.C., YU, G., and BROWN, P.O. (1993). Integration of murine leukemia virus DNA depends on mitosis. EMBO J. 12, 2099-2108.
33. SCHLUTER, C., DUCHROW, M., WOHLENBERG, C., BECKER, M.H., KEY, G., FLAD, H.D., and GERDES, J. (1993). The cell proliferation-associated antigen of antibody Ki-67: A very large, ubiquitous nuclear protein with numerous repeated elements, representing a new kind of cell cycle-maintaining proteins. J. Cell Biol. 123, 513-522.
34. SITNICKA, E., LIN, N., PRIESTLEY, G.V., FOX, N., BROUDY, V.C., WOLF, N.S., and KAUSHANSKY, K. (1996). The effect of thrombopoietin on the proliferation and differentiation of murine hematopoietic stem cells. Blood 87, 4998-5005.
35. TAKAHASHI, T., YAMADA, K., TANAKA, T., KUMANO, K., KUROKAWA, M., HIRANO, N., HONDA, H., CHIBA, S., TSUJI, K., YAZAKI, Y., NAKAHATA, T., and HIRAI, H. (1998). A potential molecular approach to ex vivo hematopoietic expansion with recombinant epidermal growth factor receptor-expressing adenovirus vector. Blood 91, 4509-4515.
36. TISDALE, J.F., HANAZONO, Y., SELLERS, S.E., AGRICOLA, B.A., METZGER, M.E., DONAHUE, R.E., and DUNBAR, C.E. (1998). Ex vivo expansion of genetically marked rhesus peripheral blood progenitor cells results in diminished long-term repopulating ability. Blood 92, 1131-1141.
37. TSIEN, R.Y. (1998). The green fluorescent protein. Annu. Rev. Biochem. 67, 509-544.
38. WANG, J.C., DOEDENS, M., and DICK, J.E. (1997). Primitive human hematopoietic cells are enriched in cord blood compared with adult bone marrow or mobilized peripheral blood as measured by the quantitative in vivo SCID-repopulating cell assay. Blood 89, 3919-3924.
39. WATANABE, T., KUSZYNSKI, C., INO, K., HEIMANN, D.G., SHEPARAD, H.M., YASUI, Y., MANEVAL, D.C., and TALMADGE, J.E. (1996). Gene transfer into human bone marrow hematopoietic cells mediated by adenovirus vectors. Blood 87, 5032-5039. [See comments]
40. WICKHAM, T.J., MATHIAS, P., CHERESH, D.A., and NEMEROW, G.R. (1993). Integrins alpha v beta 3 and alpha v beta 5 promote adenovirus internalization but not virus attachment. Cell 73, 309-319.